Bay Area drugmaker under pressure to allow “compassionate use” of experimental therapy

A 45-year-old Austin woman who’s been battling ovarian cancer for the past seven years is using social and mainstream media to lobby a San Rafael pharmaceutical company to give her immediate access to an experimental drug she believes will be her best chance for survival.

The company, BioMarin, has refused Sloan what’s known as a “compassionate use” exception, or the use of a non-approved therapies by patients who have life-threatening diseases and have exhausted all available and approved options, including clinical trials.

On its face, the story sounds similar to the case last year of a Florida mom who made a similar plea to South San Francisco drugmaker Genentech for an unapproved breast cancer drug called pertuzamab. Darlene Gant’s video went viral last April and within days of seeing the video, Genentech officials agreed to provide her with the drug.

Meanwhile, BioMarin’s drug, BMN 673, just completed small, early-stage trials involving 28 ovarian cancer patients and is currently recruiting breast cancer patients for a later-stage study. Company officials said they hoped to help get Sloan enrolled in another trial, but said extending compassionate use for this drug would be “premature.”

“Although the current data from BMN 673 that we have looks promising, there is no data at this point to support anything beyond dosing and some preliminary safety,” the company said in a statement. “It is too early to know if the experimental therapy is safe or effective, or will even prolong life.”

These cases are frustrated, complicated and heartbreaking, but when should a drugmaker allow the use of its investigational therapies outside the trial process?

Sloan, an attorney who heads the Texas Advocacy Project, which provides free legal services for victims of domestic and sexual violence, doesn’t have the time to wait. Sloan and her supporters have initiated an aggressive lobbying campaign to win access and have started an online petition.